SMN2 splicing modulator TEC-1

SKU: orb1818861

Description

SMN2 splicing modulator TEC-1 is a novel specific, CNS penetrant small molecule SMN2 splicing modulator, increases the expression level of FL-SMN2 mRNA and decreases the expression level of Δ7 mRNA.TEC-1 showed higher selectively (>60-fold) on galactosylceramidase and huntingtin gene expression compared to previously reported compounds (e.g., SMN-C3) due to off-target effects on cryptic exon inclusion and nonsense-mediated mRNA decay.TEC-1 modulates SMN2 splicing and displays disease-modifying effects in motor neurons derived from SMA patient iPSCs (GM24468).|TEC-1 rescues the phenotype in a murine model of spinal muscular atrophy (SMA).

Key Properties

• CAS Number: 2407723-70-8
• MW: 389.463
• Purity: >98%
• Formula: C₂₁H₂₃N₇O

Storage & Handling

• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only